Pipeline
There are currently three focus areas which form the portfolio and pipeline of Helix Therapeutics. Two projects target the human globin gene. Specific, well defined and distinct mutations in this gene lead to sickle cell disease and beta thalassemia. The repair technology has been successfully used to modify the beta-globin gene in human primary CD34+ hematopoietic stem cells and demonstrate that these modified cells give rise to erythroid lineages that express red blood cell markers and retain the beta-globin gene modification, (Chin et al. 2008. PNAS; 105(36):13514-9).
The CCR5 project addresses a very exciting area of research in HIV/AIDS. A receptor on the surface of T cells, CCR5, is required for HIV entry into cells. Helix Therapeutics has introduced a modification into the CCR5 gene of human hematopoietic stem cells which renders the cells resistant to HIV entry and demonstrated the expression of the modified receptor on the cell surface.
The third focus area addresses a class of genetic diseases which give rise to lysosomal storage disorders. Perhaps the best known of these diseases is Gaucher’s disease in which macrophages are unable to process glucocerebroside, resulting in multi-organ dysfunction. A mutation in a different metabolizing enzyme causes Hurler Syndrome, also a devastating disease. Early work is ongoing to design TFO reagents to correct the responsible mutations.
